Novartis announced that the U.S. Food and Drug Administration (FDA) approved Fabhalta (iptacopan) as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH)1. Fabhalta is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system, providing comprehensive control of red blood cell (RBC) destination within and outside the blood vessels (intra- and extravascular hemolysis [IVH and EVH). In clinical trials, treatment with Fabhalta increased hemoglobin levels (2 g/dL from baseline in the absence of RBC transfusions) in the majority of patients and in APPLY-PNH nearly all patients treated with Fabhalta did not receive blood transfusions1-5. The FDA approval is based on the Phase III APPLY-PNH trial in patients with residual anemia (hemoglobin < 10 g/dL) despite prior anti-C5 treatment who switched to Fabhalta, which demonstrated superiority in hemoglobin improvement in the absence of RBC blood transfusions and in APPLY- PNH nearly all patients treated with FabH did not receive blood transfusions. Approval was also supported by the Phase III APPOINT-PNH study in complement inhibitor-naive patients.
The 24-week core treatment periods in APPLy-PNH and APPOINT-PNH trials respectively showed1-3: Patients with sustained increase of hemoglobin levels 2 g/dLa from baseline in the absence ofRBC transfusions: 82.3% of anti-C5-experienced Fabhalta patients responded vs. 0% for anti-C5 It is important for patients to show this card to any doctor who treats them. This will help doctors diagnose and treat patients quickly.
FABHALTA is only available through a program called the FABHALTA Risk Evaluation and Mitigation Strategy (REMS). Before patients can take FABHALTA, their doctor must enroll in the FABHALTA REMS program, counsel patients about the risk of serious infections caused by certain bacteria, give patients information about the symptoms of serious infections, make sure that patients are vaccinated against serious infections caused by encapsulated bacteria and that they receive antibiotics if they need To treat patients.